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Europe’s first gene therapy approval anticipated

WHAT a milestone. After decades of development, regulators have recommended approval for the first commercial gene therapy in Europe and the US.

The therapy is for a rare genetic condition called lipoprotein lipase deficiency. People with LPLD can’t break down fat globules in the bloodstream, known as chylomicrons. They lack the necessary enzyme, lipoprotein lipase, which is normally made in muscle cells. Because of defects in the gene for the enzyme, fat clogs up blood vessels in the gut and pancreas of those with LPLD. So they have to avoid all fat in food.

The new therapy, called Glybera, is produced by uniQure BV in the Netherlands. It involves injecting a virus containing a healthy copy of the gene into muscle cells in a patient’s leg.

On 20 July, the European Medicines Agency recommended that Glybera be approved. The European Commission will make the final decision within three months – however, it usually follows the EMA’s advice.